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New gene therapy hope for one of the most lethal childhood cancers

17 November 2010

Scientists at the UCL Institute of Child Health (ICH) have developed a new gene therapy that could have the potential to save the lives of children with a life threatening tumour called neuroblastoma. The technique, which uses novel tumour-homing nanoparticles has proved to be effective in a first stage trial in which researchers successfully targeted the tumours in a mouse model.

The details of the study are published online today in the international journal Biomaterials.

Stephen Hart, reader in molecular genetics at the ICH, explains: “It has long been a major technical challenge for medical researchers to use gene therapy to target this type of tumour, particularly when the cancer has spread. Now with the development of these novel nanoparticles in our laboratory, we’ve been able to deliver the genes to where they are needed, via an intravenous injection.”

Neuroblastoma is one of the most aggressive malignancies, affecting around 100 children each year in Britain.  New treatments are urgently needed to tackle the disease, which is often fatal. Two thirds of children have widespread disease at diagnosis, making treatment even more challenging for specialist clinicians.

“In the mouse tumour model we have demonstrated that the nanoparticles can home in on tumours after injection into the blood stream, avoiding the liver, lung and spleen, organs that might otherwise remove the particles from the circulation. We have then used the nanoparticles to deliver a cargo of anti-tumour genes, which in turn stimulated the mouse’s immune cells to attack and destroy the tumour. We observed that tumour growth was slowed significantly and in a third of mice, tumours were eradicated completely, surviving long-term.”

“These nanoparticles are composed of peptides (small pieces of protein) and liposomes (fatty globules), as well as the therapeutic genes. Although similar to artificial viruses, the nanoparticles are safe and non-infectious.”

Dr Penelope Brock, consultant oncologist at Great Ormond Street Hospital said: “This is an extremely exciting breakthrough with enormous promise for improving clinical care of children and adolescents suffering from a very aggressive disease. I look forward to seeing results of early phase clinical trials.”

Dr Hart continues, “We now need to study the efficacy, safety and side-effects of the nanoparticles and hope that in the future our findings will translate into a viable treatment for some of the most challenging cases.”

Contact information:

For further information please contact Hayley Dodman, Great Ormond Street Hospital press office on 020 7239 3126 or email dodmah@gosh.nhs.uk

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Notes to editors

Great Ormond Street Hospital for Children NHS Trust is the country’s leading centre for treating sick children, with the widest range of specialists under one roof.

With the UCL Institute of Child Health, we are the largest centre for paediatric research outside the US and play a key role in training children’s health specialists for the future.

Our charity needs to raise £50 million every year to help rebuild and refurbish Great Ormond Street Hospital, buy vital equipment and fund pioneering research. With your help we provide world class care to our very ill children and their families.